Muscular Atrophy News and Research

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Spark Therapeutics launches gene therapy clinical trial for late-onset Pompe disease

Pompe disease is a hereditary genetic disorder caused by a deficiency of acid alpha-glucosidase leading to build-up of glycogen in the lyosomes, which then causes cell damage in various tissues, in particular the heart, the muscles, the liver and the nervous system.

3 Feb 2021

Canadian Neuromuscular Disease Registry increases patient access to research, clinical trials

The Canadian Neuromuscular Disease Registry (CNDR) was launched in 2010 to increase efficient patient access to cutting-edge research and clinical trials, to increase understanding of the natural history and epidemiology of neuromuscular disease across Canada, and to facilitate research collaboration.

3 Feb 2021

Designer DNA agent reduces myeloma stem cell abundance, lowers disease burden in mice

Many patients with multiple myeloma, a type of blood cancer, eventually develop resistance to one treatment after another. That's in part because cancer stem cells drive the disease -- cells that continually self-renew. If a therapy can't completely destroy these malignant stem cells, the cancer is likely to keep coming back.

20 Jan 2021

Replacing the RNA strand with DNA may enhance efficacy of ASO-based drugs

Antisense oligonucleotides (ASO) hold great promise for pharmacotherapy. Now, researchers at Tokyo Medical and Dental University (TMDU) and Ionis Pharmaceuticals, advancing their earlier work on a heteroduplex oligonucleotide (HDO) model, have demonstrated augmentation of ASO-based drugs by replacing the RNA strand with DNA.

5 Jan 2021

Amilorides reduce SARS-CoV-2 viral replication in vitro

A new U.S. study published on the bioRxiv preprint server reports the use of an amiloride-based scaffold that targets a viral RNA structure known to be essential in viral replication in the 5’-untranslated region (UTR) of the virus. The researchers say this heralds the application of these molecules as chemical probes to explore viral biology and produce specific antivirals targeting RNA regions of the coronavirus genome.

8 Dec 2020

Genethon to start international clinical trial for treatment of Duchenne muscular dystrophy

Genethon, dedicated to designing and developing gene therapy products for rare diseases, received this Monday 30th of November the authorization from the ANSM, the French National Agency for Medicines and Health Products Safety, to start in France a multicentre international clinical trial for the treatment of Duchenne muscular dystrophy with product GNT 004.

2 Dec 2020

Researchers develop telomerase gene therapy for treating different pathologies

After several years of research and many joint scientific publications, molecular biologists Maria Blasco (National Cancer Research Centre, CNIO) and Fàtima Bosch (Universitat Autònoma de Barcelona, UAB) have developed a telomerase gene therapy for the treatment of different pathologies related to shortening telomeres and, therefore, ageing.

2 Dec 2020

Study finds how the brain 're-wires' after neurological disease

Trinity College researchers are studying how the brain re-wires itself in neurological disease.

24 Nov 2020

Study implicates new gene as driver of motor neuron diseases

Failures in a quality control system that protects protein-building fidelity in cells can lead to motor neuron degeneration and related diseases, according to a new study from an international team co-directed by Scripps Research molecular biologist Claudio Joazeiro, PhD.

16 Sep 2020

Researchers find link between dystrophic muscles and lymphatic system in mice with Duchenne disease

Researchers at the Universities of Maynooth and Bonn discover a new connection in muscular dystrophy Duchenne muscular dystrophy (DMD) is the most common muscle disease in children and is passed on by X-linked recessive inheritance.

27 Aug 2020

Study examines safety and early outcomes of gene therapy for spinal muscular atrophy

In May 2019, the U.S. Food and Drug Administration approved a gene replacement therapy for the inherited, progressive neuromuscular disease 5q-linked spinal muscular atrophy.

25 Aug 2020

FDA approves first oral drug for treatment of spinal muscular atrophy

The U.S. Food and Drug Administration today approved Evrysdi (risdiplam) to treat patients two months of age and older with spinal muscular atrophy (SMA), a rare and often fatal genetic disease affecting muscle strength and movement.

7 Aug 2020

Researchers develop new therapeutic approach to treat CLN3 Batten disease

Researchers led by a team at Rosalind Franklin University of Medicine and Science have devised a new therapeutic approach to treating a rare but deadly neurodegenerative genetic disease in children.

31 Jul 2020

C-Path gets positive response from FDA for drug-induced skeletal muscle injury safety biomarkers

Critical Path Institute announced today that the Biomarker Qualification Program at the Center for Drug Evaluation and Research in the U.S. Food and Drug Administration issued a positive response to the Letter of Intent developed by C-Path's Predictive Safety Testing Consortium and Duchenne Regulatory Science Consortium, for a panel of four safety biomarkers of acute drug-induced skeletal muscle injury.

23 Jul 2020

New editorial published following adverse events of gene therapy clinical trial

The Editor-in-Chief of Human Gene Therapy, the first journal devoted to the field of gene therapy, and one of the world's leading experts on gene therapy have co-authored a new editorial, Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy, in response to the news of two deaths in a now-halted gene therapy clinical trial.

3 Jul 2020

Developing Vaccines and Therapeutics by Exploiting Structure in Spherical Nucleic Acids

An interview discussing the use of spherical nucleic acids (SNAs) and what they show about the importance of structure in pharmaceutical development.

From Pittcon 2 Jul 2020

Researchers develop a potential treatment approach for Pelizaeus-Merzbacher disease

A team led by Case Western Reserve University medical researchers has developed a potential treatment method for Pelizaeus-Merzbacher disease (PMD), a fatal neurological disorder that produces severe movement, motor and cognitive dysfunction in children.

1 Jul 2020

Researchers identify new molecules to study specific glial cells integral to synapses

While the brain is composed of two types of cells, glia tend to receive far less attention for their importance in brain function and disease than the more celebrated neurons.

26 Jun 2020

New therapeutic approach for Parkinson’s and other neurodegenerative diseases

Xiang-Dong Fu, PhD, has never been more excited about something in his entire career. He has long studied the basic biology of RNA, a genetic cousin of DNA, and the proteins that bind it. But a single discovery has launched Fu into a completely new field: neuroscience.

25 Jun 2020

German researchers recommend early screening and treatment for spinal muscular atrophy

The advent of therapeutic interventions for spinal muscular atrophy (SMA) has increased the importance of presymptomatic diagnosis and treatment. When to start treatment in children with less severe disease remains controversial.

11 Jun 2020